An observational study in Gaucher Disease, sponsored by Shire. Recruiting at 1 site in United States. Per ClinicalTrials.gov, last updated 2026-09-15.
Sponsored by Shire · Observational
The Gaucher Outcomes Survey (GOS) is an ongoing observational, international, multi-center, long-term Registry of Patients with Gaucher Disease irrespective of their treatment status or type of treatment received. No experimental intervention is involved. Patients undergo clinical assessments and receive care as determined by the patients' treating physician.
The objectives of the registry include to evaluate the safety and long-term effectiveness of velaglucerase alfa, to characterize patients receiving velaglucerase alfa or other Gaucher Disease-specific treatments, to gain a better understanding of the natural history of GD and to serve as a database for evidence-based management of Gaucher Disease over time in real-life clinical practice.
20 MAY 2020: The temporary enrollment stop of new patients into this study due to the COVID-10 pandemic has been lifted in one or more countries/sites, and the study is now again enrolling new patients. However, some countries/sites may still have paused the enrollment of new patients due to the pandemic.
24 APRIL 2020: Enrollment of new patients into this study has been paused due to the COVID-19 situation. The duration of this pause is dependent on the leveling and control of the COVID-19 pandemic.
GOS is a disease specific registry irrespective of treatment or treatment status, open to all patients of any age or sex with Gaucher disease of any type. Patients included may be those who are untreated, naive to therapy, individuals who are currently or have been previously treated with velaglucerase alfa (VPRIV), or individuals who have been receiving or are currently exposed to other treatments for Gaucher disease. There is no predefined sample size.
Exclusion Criteria:
- Patients currently enrolled in ongoing blinded clinical trials (drugs or devices; includes all blinded trials)
GOS is a disease specific registry open to all Gaucher patients irrespective of treatment status or type of treatment
Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Treatment-emergent adverse events (TEAEs) are defined as adverse events (AEs) that either commenced or worsened following the first dose of VPRIV.
Time frame: Baseline to one year for up to 20 years
Number of Participants With Infusion-related Reactions (IRRs)
An IRR is defined as an AE that has been assessed as at least possibly related to treatment with VPRIV and occurs during an infusion or up to 24 hours post-VPRIV infusion.
Time frame: Baseline to one year for up to 20 years
Increase of Hemoglobin Concentration
Hemoglobin concentration will be assessed.
Time frame: Baseline to one year for up to 20 years
Increase of Platelet Count
Platelet count will be assessed.
Time frame: Baseline to one year for up to 20 years
Decrease in Liver Volume
Liver volume will be assessed by abdominal imaging.
Time frame: Baseline to one year for up to 20 years
Decrease in Spleen Volume
Spleen volume will be assessed by abdominal imaging.
Time frame: Baseline to one year for up to 20 years
Increase in Bone Mineral Density (BMD)
Bone mineral density will be assessed.
Time frame: Baseline to one year for up to 20 years
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
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